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Journal Articles

Discovery of the transcription factor ZNF683/hobit as a central regulator of pathogenic T cell proliferation in acute gvhd

Alexandre Albanese, Edward Chen, Yvonne Suessmuth, Jiayi Dong, James Kaminski, Lev Gorfinkel, Ally Eran, Jillian Zavistaski, Lorenzo Cagnin, Paul Keskula, Brandi Bratrude, Kayla Betz, Elham Rayzan, Muna Qayed, John Horan, Amelia Langston, Benjamin Watkins, Bruce Blazar, Leslie Kean

Blood (2025) 146 (Supplement 1): 275.

https://doi.org/10.1182/blood-2025-275

Published: 2025

Journal Articles

In Vivo correction of the sickle cell disease mutation in hematopoietic stem cells using RNA gene writers

Blood (2025) 146 (Supplement 1): 2536.

https://doi.org/10.1182/blood-2025-2536

Published: 2025

Journal Articles

T cell receptor costimulation blockade effectively prevents acute graft versus host disease without reducing the size or diversity of the T cell immune repertoire

Chiara Wychera, Alexandre Albanese, Steven Siegel, Joseph Schmalz, Stephanie Bien, Brandi Bratrude, Kayla Betz, Lev Gorfinkel, Lorenzo Cagnin, Paul Keskula, Bruce Blazar, Muna Qayed, John Horan, Amelia Langston, Benjamin Watkins, Leslie Kean

Blood (2025) 146 (Supplement 1): 819.

https://doi.org/10.1182/blood-2025-819

Published: 2025

Journal Articles

Refined design of a CD117 antibody-drug conjugate safely and effectively conditions non-human primates for autologous transplant-based gene therapy

Blood (2025) 146 (Supplement 1): 5871.

https://doi.org/10.1182/blood-2025-5871

Published: 2025

Journal Articles

A first-in-primate In Vivo CRISPR/Cas9 screen in a rhesus macaque model identifies drivers of CAR-T expansion, tissue tropism and persistence

Francesca Alvarez Calderon, Marlana Winschel, Smriti Srikanth, Marc Schwartz, Ryan Fleming, Lev Gorfinkel, Rachel Hu, Casey Son, John Doench, Leslie Kean, Ulrike Gerdemann

Blood (2025) 146 (Supplement 1): 2329.

https://doi.org/10.1182/blood-2025-2329

Published: 2025

Journal Articles

Children and adolescents with sickle cell disease demonstrate improved health-related quality of life and low decisional regret after hematopoietic cell transplantation: A sickle cell transplant advocacy and research alliance (STAR) study

Blood (2025) 146 (Supplement 1): 4278.

https://doi.org/10.1182/blood-2025-4278

Published: 2025

Journal Articles

A single dose of a CD137 antibody–drug conjugate protects rhesus macaque allogeneic HCT recipients against acute GVHD

Blood (2025) 146 (9): 1127–1141.

https://doi.org/10.1182/blood.2024027239

Published: 2025

Includes: Supplemental data

Journal Articles

ANKRD26-related thrombocytopenia 2 with a baseline increase in blasts: implications for clinical surveillance

Lara Wahlster, Lucy A. Godley, Jason X. Cheng, Michael W. Drazer, Christopher R. Reilly, Geraldine S. Pinkus, Jared H. Rowe, Nina Weichert-Leahey, R. Grant Rowe, Lev Gorfinkel, Karyn Brundige, Franziska Wachter, Luana Messa, Alan D. Michelson, Alan B. Cantor, Akiko Shimamura, Jacob R. Bledsoe

Blood (2025) 146 (2): 254–259.

https://doi.org/10.1182/blood.2024027368

Published: 2025

Includes: Supplemental data

Journal Articles

Five-Year Outcomes of the “Abatacept Combined with a Calcineurin Inhibitor and Methotrexate for Graft Versus Host Disease (GVHD) Prophylaxis: A Randomized Controlled Trial” (‘ABA2‘)

Blood (2024) 144 (Supplement 1): 3516.

https://doi.org/10.1182/blood-2024-205130

Published: 2024

Journal Articles

Gene Therapy for Sickle Cell Disease Is Associated with a Decreased Transplant Length of Stay and Similar Rates of Short-Term Complications Compared to Allogeneic Matched Sibling Stem Cell Transplant: A Single Center Experience

Lev Gorfinkel, Erica B. Esrick, Alexander Lake, Miki Nishitani, Malika Kapadia, Amy Federico, Colleen Dansereau, David A. Williams, Leslie E. Lehmann

Blood (2024) 144 (Supplement 1): 3574.

https://doi.org/10.1182/blood-2024-198677

Published: 2024

Journal Articles

Alloengraftment without significant toxicity or GVHD in CD45 antibody-drug conjugate–conditioned Fanconi anemia mice

Blood (2024) 143 (21): 2201–2216.

https://doi.org/10.1182/blood.2023023549

Published: 2024

Includes: Supplemental data

Journal Articles

Enhanced T Cell Function of PTPN2 Deleted CAR-T Cells Comes at a Cost: PTPN2 Knockout CAR-Ts Secrete More Cytokines and Demonstrate Increased Cytotoxicity, but Exhibit More Severe CRS and Icans in a Non-Human Primate Model

Francesca Alvarez Calderon, Ryan A Fleming, Lev Gorfinkel, Katherine Michaelis, Xianliang Rui, James Kaminski, Dana Bonin, Anick Mallette, Katherine Brodeur, Heber Domingues, Jennifer Lane, Victor Tkachev, Leslie Kean, Ulrike Gerdemann

Blood (2023) 142 (Supplement 1): 2087.

https://doi.org/10.1182/blood-2023-187499

Published: 2023

Journal Articles

In Vivo Delivery of RNA By Lipid Nanoparticles (LNP) to Hematopoietic Stem Cells and T Cells in Humanized Mice and Non-Human Primates

Blood (2023) 142 (Supplement 1): 4818.

https://doi.org/10.1182/blood-2023-187271

Published: 2023

Journal Articles

Abatacept for the Prevention of Gvhd in Pediatric and Adult Patients Receiving 7/8 HLA-Mismatched Unrelated Transplant for Hematologic Malignancies: A Real-World Analysis

Sharmila Raghunandan, Lev Gorfinkel, Michael Graiser, Brandi Bratrude, Kayla Betz, Yvonne Suessmuth, Scott Gillespie, Adrianna Lynn Westbrook, Kirsten M. Williams, Michelle Schoettler, Amelia Langston, Leslie Kean, Muna Qayed, John T. Horan, Benjamin Watkins

Blood (2022) 140 (Supplement 1): 7653–7655.

https://doi.org/10.1182/blood-2022-163627

Published: 2022

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Discovery of the transcription factor ZNF683/hobit as a central regulator of pathogenic T cell proliferation in acute gvhd

In Vivo correction of the sickle cell disease mutation in hematopoietic stem cells using RNA gene writers

T cell receptor costimulation blockade effectively prevents acute graft versus host disease without reducing the size or diversity of the T cell immune repertoire

Refined design of a CD117 antibody-drug conjugate safely and effectively conditions non-human primates for autologous transplant-based gene therapy

A first-in-primate In Vivo CRISPR/Cas9 screen in a rhesus macaque model identifies drivers of CAR-T expansion, tissue tropism and persistence

Children and adolescents with sickle cell disease demonstrate improved health-related quality of life and low decisional regret after hematopoietic cell transplantation: A sickle cell transplant advocacy and research alliance (STAR) study

A single dose of a CD137 antibody–drug conjugate protects rhesus macaque allogeneic HCT recipients against acute GVHD

ANKRD26-related thrombocytopenia 2 with a baseline increase in blasts: implications for clinical surveillance

Five-Year Outcomes of the “Abatacept Combined with a Calcineurin Inhibitor and Methotrexate for Graft Versus Host Disease (GVHD) Prophylaxis: A Randomized Controlled Trial” (‘ABA2‘)

Gene Therapy for Sickle Cell Disease Is Associated with a Decreased Transplant Length of Stay and Similar Rates of Short-Term Complications Compared to Allogeneic Matched Sibling Stem Cell Transplant: A Single Center Experience

Alloengraftment without significant toxicity or GVHD in CD45 antibody-drug conjugate–conditioned Fanconi anemia mice

Enhanced T Cell Function of PTPN2 Deleted CAR-T Cells Comes at a Cost: PTPN2 Knockout CAR-Ts Secrete More Cytokines and Demonstrate Increased Cytotoxicity, but Exhibit More Severe CRS and Icans in a Non-Human Primate Model

In Vivo Delivery of RNA By Lipid Nanoparticles (LNP) to Hematopoietic Stem Cells and T Cells in Humanized Mice and Non-Human Primates

Abatacept for the Prevention of Gvhd in Pediatric and Adult Patients Receiving 7/8 HLA-Mismatched Unrelated Transplant for Hematologic Malignancies: A Real-World Analysis

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